Read the full article by Toni Morrissey at here.

Madison, Wisconsin – A unique new therapy for children and young adults with a particular form of leukemia received Food and Drug Administration (FDA) approval today. American Family Children’s Hospital will be one of a handful of certified treatment centers nationwide that offer the treatment, another example of personalized medicine.

Known as chimeric antigen receptor (CAR)-T cell therapy, Kymriah (tisagenlecleucel) was approved to treat acute lymphoblastic leukemia (ALL) that has resisted other treatment or has relapsed a second time. CAR-T cell therapy engineers a child’s immune cells (called T-cells) to express a CAR to attach to and eliminate those leukemia cells that express a specific antigen on their cell surface. The extracted T-cells are sent to a processing center where they are reprogrammed to express the receptor, and then shipped back to American Family Children’s Hospital for infusion into the child with relapsed leukemia. The collection of the patient’s T-cells and the infusion of the CAR-T cells are both outpatient procedures.

American Family Children’s Hospital was one of a small number of sites that conducted clinical trials for CAR-T cell therapy. One patient has been in “deep remission” for several months, according to site principal investigator Dr. Christian Capitini, a pediatric hematologist/oncologist at UW Health.

“Our patient had run out of options except for this clinical trial,” said Capitini. “After all the patient has been through, it’s amazing to say she is now in remission.”

The infusion has been associated with unique side effects, including cytokine release syndrome (CRS) and neurotoxicity. These side effects are usually managed in the inpatient setting. CRS can range from high fevers and body aches, to life-threatening problems such as maintaining safe blood pressures and need for a ventilator. CRS can be treated with drugs, and today the drug Actemra (tocilizumab) was given an expanded approval to help treat CRS. Afterward, follow-up care involves an outpatient monthly immunoglobulin infusion to prevent infections because all normal B-cells are eliminated by the therapy.

The therapy originally received the FDA’s Fast Track, Priority Review and Breakthrough Therapy designations in July, 2014. Last month, an FDA advisory committee unanimously recommended approval of the therapy. Final approval was given today.